Breakthrough Study Finds Six Children Regain Hearing
A groundbreaking study published on Wednesday revealed that children with hereditary deafness regained their hearing through a revolutionary gene therapy. Led by researchers from Mass Eye and Ear, a specialist hospital in Boston, the clinical trial focused on six children with a genetic form of deafness known as DFNB9.
Understanding the Cause of Deafness
The deafness in these children stemmed from mutations in the OTOF gene, which prevents the production of a crucial protein called otoferlin. This protein is vital for transmitting sound signals from the ear to the brain, as explained by the researchers.
Success of the Trial
The trial, conducted at Eye & ENT Hospital of Fudan University in Shanghai, involved the use of an inactive virus carrying a functional version of the OTOF gene. Administered at varying doses directly into the inner ear of the children, the results after 26 weeks were nothing short of remarkable.
Five out of the six children, previously classified as having total deafness, not only regained their hearing but also regained the ability to engage in normal conversations.
Impact and Future Prospects
Described as a milestone by Dr. Zheng-Yi Chen from Mass Eye and Ear, this pioneering gene therapy marks the first human clinical trial for treating this type of genetic deafness. Chen emphasized the significance of the treatment in restoring hearing function, especially in children, where hearing loss can significantly impact their development.
“This is the first time a brand-new gene therapy approach has been successful in treating children who are completely deaf and restoring their hearing function,” Chen stated.
Expanding the Study
The next steps involve expanding the study to enroll more patients for a larger clinical trial. The goal is to monitor the long-term efficacy of the treatment to potentially extend its application to various types of genetic deafness.
The findings from this groundbreaking clinical trial will be presented at the Association for Research in Otolaryngology Annual Meeting in California. This development follows the recent report of a successful gene therapy treatment in an 11-year-old boy in the U.S., showcasing the promise of gene therapy in reversing genetic forms of hearing loss.
